Researchers at the Department of Health and Human Services' National Institutes of Health (NIH) have launched a five-year study to see whether a therapy using transplantation of hematopoietic stem cells, blood stem cells found in bone marrow, can produce long-term remission for patients with severe, treatment-resistant systemic lupus erythematosus (or lupus), a rheumatic autoimmune disease that can affect the body's major organs. The study will include a basic research component to examine the roles of B and T cells, white blood cells in the immune system, in triggering lupus symptoms.
In this pilot study, 14 patients from ages 15 to 40 will receive stem cell transplantation therapy, during which their stem cells will be removed from their bone marrow. These cells, which will become different kinds of blood and immune system cells in the body, will then be harvested and cleaned. After the patient's bone marrow is treated with immunosuppressant drugs to destroy the disease-causing immune cells, the stem cells will be returned to the bone marrow. The stem cells will then repopulate the marrow and body to establish a more properly functioning immune system.
The initial treatment requires several outpatient visits followed by a two-week hospital stay. Patients then have monthly medical visits for six months, quarterly visits for two years, and annual visits for the remainder of the study. Following therapy, researchers will assess whether this treatment produces sustained, relapse-free disease remission for 24 months.
"Many patients with severe forms of lupus have limited treatment options that may offer only temporary relief of symptoms and no disease regression. For these patients, stem cell transplantation therapy may offer hope for a normal functioning immune system," said Stephen I. Katz, M.D., Ph.D., director of NIH's National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).
Severe forms of lupus can devastate patients, causing pain, fatigue, depression, and in some cases, premature death. Patients who enter this study must have been treated, to no avail, with high doses of immunosuppressant drugs, which decrease immune function. Researchers believe that by combining immunosuppressant treatment with stem cell transplantation, they can create a new immune system that doesn't attack the body's healthy cells.
"Advances in cancer therapy can yield advances in treatment of autoimmune diseases," said Andrew von Eschenbach, M.D., director of the NIH's National Cancer Institute. "Cyclophosphamide used as a cancer therapy suppresses the immune system. This discovery led to improvements for lupus patients. Similarly, altering the immune system through progenitor cell transplantation, a cancer treatment, may also benefit lupus patients."
"Lupus is different from cancer," he added. "However, we have found that both diseases can benefit from treatment that modulates the immune system."